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Statistical Power in Quantitative Diffusion MRI of Tumor Response

Rationale and Objectives

Diffusion magnetic resonance imaging may be useful in tracking tumor growth and response to treatment. However, studies using these measures may lack statistical power to draw definitive conclusions regarding changes in tumor cellularity. Using apparent diffusion coefficient values taken from the literature, the investigators estimated sample sizes for a range of changes to the mean.

Materials and Methods

A literature search was performed of studies measuring the average apparent diffusion coefficients for various bodily tissues, and the mean and standard deviation from each study were recorded. Analyses of statistical power were then performed using these values and comparing them to a population of healthy controls.

Results

Tumor cellularity as measured by apparent diffusion coefficients may have high sensitivity, but the analyses indicate that investigations in this field may potentially suffer from low statistical power. For example, the findings indicate that samples of <20 patients may require a mean change of approximately 25% between study conditions.

Conclusions

Suggestions are offered for improvements in methodologic approaches and in data reporting to assist in overcoming the limitations of small sample sizes. On the basis of this literature review, reference values are provided to help investigators estimate study sample size to achieve adequate statistical power.

Advances in magnetic resonance imaging (MRI) have provided new means of tracking tumor progression and response to treatment. In particular, diffusion-weighted imaging, which tracks the microscopic rate of water diffusion within tissues, may hold many advantages over traditional anatomic MRI techniques by providing information about tissue cellularity and the integrity of cell membranes . Recent research has focused on the apparent diffusion coefficient (ADC), which is reduced as cell concentration increases and is more sensitive to changes in tumor progression or response to treatment than traditional measures, such as tumor volume . ADC values may potentially predict tumor grade and response to therapy, possibly because of the inherent barrier to water diffusion of tumor cells . Numerous studies have found large changes in ADCs in response to chemoradiation , stereotactic irradiation , convection-enhanced delivery of therapeutic agents , and other therapies . Generally, increases in ADC values after therapy are associated with a positive treatment response, which may be due to decreased cell proliferation and density and an increased extracellular space from cell shrinkage during apoptotic cell processes seen in a positive therapeutic response . In addition to changes in ADCs over time and among patient groups, it has also been noted that intratumoral ADC may be valuable, because areas of tumor in which the ADC is stable may represent persistent disease . Additionally, minimum intratumoral ADC values have been shown to have clinical relevance, as studies on human malignant gliomas have shown a correlation between minimum ADC values with higher tumor grade and shorter survival times .

Given these findings, ADC and other diffusion parameters may have a significant role in noninvasively quantifying response to treatment, guiding treatment plans, and research. However, measurements of ADC are highly variable across patients, and many studies are underpowered to detect small or moderate changes in ADC during the course of therapy. This may lead to the selective publication of research that identifies large effects of interventions on ADCs, while subtler ADC changes may be lost as false-negatives. Challenges facing the application of diffusion-weighted imaging have only recently begun to be explored. For example, Heusner et al found diffusion imaging to be a sensitive, though relatively nonspecific, diagnostic tool in identifying metastatic breast tumors. Considering work in rodents that has demonstrated the high sensitivity of ADC to subcellular changes in tumor pathology , it is probable that small changes in ADC are indeed present but have been missed by studies with low power.

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Materials and methods

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n=2(z1−α/2+z1−β)2(μ0−μ1σ)2 n

=

2

(

z

1

α

/

2

+

z

1

β

)

2

(

μ

0

μ

1

σ

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2

An additional analysis was performed using mean and SD values for ADC gathered by an independent meta-analysis for head and neck cancer reported by Vandecaveye et al . In this analysis of the literature, the mean and SD of ADC were calculated to be 0.99 × 10 −3 and 0.26 × 10 −3 mm 2 /s, respectively, for lymph nodes that were negative on biopsy.

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Results

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Figure 1, Analyses based on review of the literature. Using values of mean and standard deviation for apparent diffusion coefficient taken from the literature, the relationship between sample size and changes to the mean is illustrated for the conservative statistical power of 0.8. Note the nonlinear relationship between the two variables, which indicates that small changes in the mean require large increases in sample size, particularly when the effect is small.

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Figure 2, Analyses based on apparent diffusion coefficient (ADC) measures from a healthy control population. Analyses depicted in Figure 1 are again represented, but these data were taken from an independent population of healthy control subjects. The control group had lower ADC variability, which results in a dramatic improvement in statistical power relative to the patient population drawn from the literature. These results serve to quantify the increased variability and resultant drop in power in clinical studies.

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Discussion

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Supplementary Data

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Supplementary Table 1

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References

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